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Phase 2 Study of LUM-201 in Children Who Have Previously Completed the LUM-201-01 Trial (OraGrowtH213) (OraGrowtH213)

Primary Purpose

Growth Hormone Deficiency

Status
Enrolling by invitation
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
LUM-201
Sponsored by
Lumos Pharma
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Growth Hormone Deficiency focused on measuring LUM-201, PGHD, Height, Oral

Eligibility Criteria

3 Years - 13 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Parent/caregiver must sign the informed consent, and the subject must sign the assent, as applicable.
  • Must have previously completed 12 months of daily rhGH treatment as part of the LUM-201-01 PGHD trial.
  • Is eligible for study participation as confirmed by the principal investigator (PI)

Exclusion Criteria:

  • Has a medical condition that, in the opinion of the PI and/or MM, adds unwarranted risk to use of LUM-201.
  • Uses any medication that, in the opinion of the PI and/or MM, can independently cause short stature or limit the response to exogenous growth factors.
  • Has planned or is receiving current long-term treatment with medications known to act as substrates, inducers, or inhibitors of the cytochrome system CYP3A4 that metabolizes LUM-201

Sites / Locations

  • Rady Children's Hospital
  • Children's National Hospital
  • Indiana University School of Medicine
  • M Health, Fairview Pediatric Specialty Clinics- Discovery Clinic
  • The Children's Mercy Hospital
  • University of Virginia Health System
  • Klinika Endokrynologii i Chorob Metabolicznych, Instytut Centrum Zdrowia Matki Polki
  • Sonomed - Centrum Medyczne
  • Klinika Endokrynologii i Diabetologii, Instytut "Pomnik Centrum Zdrowia Dziecka

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

LUM-201 (3.2 mg/kg/day)

Arm Description

Outcomes

Primary Outcome Measures

Annualized height velocity (AHV) measured as standing height with stadiometer
Annualized Height Velocity (AHV) measured as standing height with stadiometer

Secondary Outcome Measures

Incidence of adverse events in children with GHD
Number of events
GH Concentration on maintenance treatment
Serum GH concentration
Insulin-like growth factor 1
Serum concentrations of insulin-like growth factor 1
Insulin-like growth factor binding protein 3
Serum concentrations of insulin-like growth factor binding protein 3
Height standard deviation score (SDS)
Change in HT-SDS
Change in Weight
Change in Weight
Change in Weight-SDS
Change in Weight-SDS
Change in BMI
Change in BMI
Change in BMI SDS
Change in BMI SDS
Change in Bone Age
Change in bone age, measured by X-ray of left hand and wrist using Greulich & Pyle atlas
Insulin-like growth factor 1 standard deviation score (SDS)
Change in IGF-1 SDS

Full Information

First Posted
February 10, 2022
Last Updated
October 3, 2023
Sponsor
Lumos Pharma
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1. Study Identification

Unique Protocol Identification Number
NCT05250063
Brief Title
Phase 2 Study of LUM-201 in Children Who Have Previously Completed the LUM-201-01 Trial (OraGrowtH213)
Acronym
OraGrowtH213
Official Title
A Multicenter, Open-Label, Phase 2 Study to Evaluate Growth and Safety of LUM-201 Following 12 Months of Daily rhGH Treatment in Children With Idiopathic Growth Hormone Deficiency Who Have Previously Completed the LUM-201-01 Trial
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
February 18, 2022 (Actual)
Primary Completion Date
January 2024 (Anticipated)
Study Completion Date
January 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Lumos Pharma

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This is a multi-national trial. The goals of the trial are to study the growth response to LUM-201 administration in children with idiopathic growth hormone deficiency (GHD) previously treated with daily rhGH for 12 months in the LUM-201-01 trial.
Detailed Description
This trial will have one screening/baseline visit with tests to assess if subjects are eligible to start study therapy. The screening/baseline visit can coincide with the Month 12 visit in the LUM-201-01 trial. Subjects will receive the same dose of LUM-201 as a daily oral dose each morning. LUM-201 will be adjusted for body weight increments at each clinic visit, as applicable. The trial consists of 12 months of treatment. After screening, subjects will return to the clinic for 5 visits. During four of these clinic visits, safety laboratory samples will be collected.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Growth Hormone Deficiency
Keywords
LUM-201, PGHD, Height, Oral

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
LUM-201 (3.2 mg/kg/day)
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
LUM-201
Intervention Description
Administered orally once daily
Primary Outcome Measure Information:
Title
Annualized height velocity (AHV) measured as standing height with stadiometer
Description
Annualized Height Velocity (AHV) measured as standing height with stadiometer
Time Frame
Day 1 to Month 12
Secondary Outcome Measure Information:
Title
Incidence of adverse events in children with GHD
Description
Number of events
Time Frame
Day 1 to Month 12
Title
GH Concentration on maintenance treatment
Description
Serum GH concentration
Time Frame
Day 1 to Month 12
Title
Insulin-like growth factor 1
Description
Serum concentrations of insulin-like growth factor 1
Time Frame
Day 1 to Month 12
Title
Insulin-like growth factor binding protein 3
Description
Serum concentrations of insulin-like growth factor binding protein 3
Time Frame
Day 1 to Month 12
Title
Height standard deviation score (SDS)
Description
Change in HT-SDS
Time Frame
Day 1 to Month 12
Title
Change in Weight
Description
Change in Weight
Time Frame
Day 1 to Month 12
Title
Change in Weight-SDS
Description
Change in Weight-SDS
Time Frame
Day 1 to Month 12
Title
Change in BMI
Description
Change in BMI
Time Frame
Day 1 to Month 12
Title
Change in BMI SDS
Description
Change in BMI SDS
Time Frame
Day 1 to Month 12
Title
Change in Bone Age
Description
Change in bone age, measured by X-ray of left hand and wrist using Greulich & Pyle atlas
Time Frame
Month 6
Title
Insulin-like growth factor 1 standard deviation score (SDS)
Description
Change in IGF-1 SDS
Time Frame
Day 1 to Month 12

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
13 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Parent/caregiver must sign the informed consent, and the subject must sign the assent, as applicable. Must have previously completed 12 months of daily rhGH treatment as part of the LUM-201-01 PGHD trial. Is eligible for study participation as confirmed by the principal investigator (PI) Exclusion Criteria: Has a medical condition that, in the opinion of the PI and/or MM, adds unwarranted risk to use of LUM-201. Uses any medication that, in the opinion of the PI and/or MM, can independently cause short stature or limit the response to exogenous growth factors. Has planned or is receiving current long-term treatment with medications known to act as substrates, inducers, or inhibitors of the cytochrome system CYP3A4 that metabolizes LUM-201
Facility Information:
Facility Name
Rady Children's Hospital
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Facility Name
Children's National Hospital
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
Facility Name
Indiana University School of Medicine
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
M Health, Fairview Pediatric Specialty Clinics- Discovery Clinic
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55454
Country
United States
Facility Name
The Children's Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64111
Country
United States
Facility Name
University of Virginia Health System
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22908
Country
United States
Facility Name
Klinika Endokrynologii i Chorob Metabolicznych, Instytut Centrum Zdrowia Matki Polki
City
Lodz
Country
Poland
Facility Name
Sonomed - Centrum Medyczne
City
Szczecin
Country
Poland
Facility Name
Klinika Endokrynologii i Diabetologii, Instytut "Pomnik Centrum Zdrowia Dziecka
City
Warsaw
Country
Poland

12. IPD Sharing Statement

Plan to Share IPD
Undecided

Learn more about this trial

Phase 2 Study of LUM-201 in Children Who Have Previously Completed the LUM-201-01 Trial (OraGrowtH213)

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